FDA officials approved a record number of rare disease treatments last year. One groundbreaking medicine treats an inherited bone condition that causes intense pain and immobility. Another treats Fabry disease, a genetic condition that can lead to kidney failure or stroke.
Breakthroughs like these offer hope to 30 million Americans living with rare diseases. But there's still a long way to go. Scientists estimate there are 7,000 rare diseases, each of which afflicts fewer than 200,000 people. The majority of these ailments -- 95 percent -- lack a single approved treatment.